Sunday, March 15, 2009

Years ago, when I first started tracking research to cure type-1 diabetes, Peakman's clinical trial was one of the first ones that I added to my list and started following. And it was very frustrating, because nothing happened. Years went by and nothing happened. But now something has happened. The phase-I clinical trial ended, and the results have been published.

This research is based on the idea of using a modified protein to teach the body's immune system to not attack it's own beta cells. The clinical trial gave some people no protein, some a little protein, and some a lot. The patients were all people with established type-1 diabetes.

You can see the abstract here. The full study is pay-for-view:http://www3.interscience.wiley.com/journal/121536033/abstractAnd JDRF (which helped to fund the work) has a good description of it here:http://www.jdrf.org/index.cfm?fuseaction=home.viewPage&page_id=DD5ADF6D-110A-9BB5-F8684C43208F87D2Here is a great quote from JDRF's description:

Peptide immunotherapy is a type of treatment that uses small proteins to "reset" the immune system to a healthy state, much like the common allergy shot. In the case of proinsulin peptide therapy, the goal is to train the immune system to tolerate the insulin-producing beta cells that are the target of the immune response that causes type 1 diabetes.

From a safety point of view, this study was completely successful: no safety issues were found. So they can move on to phase-II trials. They hope to start those in less than 6 months. (That will be interesting in itself. My experience is that the time between ending a phase-I and starting a phase-II is always more than 9 months, so it will be interesting to see if these guys can do it quicker.)

Safety is the official goal of every phase-I clinical trial. However, most of them also try to generate some data on effectiveness of the treatment. Since they are testing minimal doses (for safety), expectations are set low, but it is still common to see some improvement. But I don't see that for this trial. I don't see any record that they looked for or found lower BG rates, lower A1Cs or less insulin usage. They did see some small changes for a 6 month period in the patient's immune system, but it is not clear what impact those changes would have.

So my overall summary for the research right now, is that their results from phase-I definitely get them to phase-II testing, but there is no data to evaluate if it is working or not. We'll need to wait for the phase-II trial to finish for that.

Wednesday, March 11, 2009

Here are some quick notes on recent progress in human trials to cure type-1 diabetes:
I'm going to post something on Peakman's recent results in the next few days.

Tolerx Adds Europe to Phase 3 'DEFEND' Trial of Otelixizumab

This phase-III human trial has been going on for some time in the US, but they recently expanded it to Europe as well. The goal here is to drug the immune system so that more beta cells survive the immune-self attack. This is a 200+ person trial.

This is the longest running phase-III clinical trial that I know of to cure type-1 diabetes. It has been going on for years, and initially the results were not promising. However, the company stuck with it (even as ownership changed hands from company to company). The most recent news still doesn't look too good to me. They had extended and expanded the trial in the hopes of getting some positive results even when the early results were not statistically significant.

However, another company, Teva, is putting in $10 million so hopefully they know more than has been released to the public, and there is good news in there, somewhere.
News article: http://uk.reuters.com/article/rbssHealthcareNews/idUKLI48804120090218

Diamyd Now has four (or five) clinical trials going at once

By my count Diamyd now has five different clinical trials going all at once:
1. They have a classic phase-III clinical trial for honeymoon diabetics which helps preserve insulin production so that patients use less insulin, and maybe no insulin at all. This being done in the US, and being 300+ patients.
2. They have a "twin" phase-III clinical trial being done in Europe with another 300+ patients.
3. They have extended their previous phase-II trial (to continue to run it for several extra years) to look for longer term effects (both good and bad).
4. They have phase-II clinical trial aimed at both preserving existing insulin production and regrowing new beta cells (a possible non-honymoon cure).
5. They have a phase-II clinical trial aimed at preventing type-1 diabetes by giving the treatment to people at high risk of type-1, but who have not shown symptoms as yet.

Osiris Therapeutics's PROCHYMAL in the news

This is the same research that Kimberly Wainscoat asked about. Osiris has been running a phase-II trial since mid last year. Their PROCHYMAL treatment has been show safe in several phase-I, II, and even III trials for several immune diseases, so they are trying it with type-1 diabetes. This is an adult (actually self) stem cell treatment. Since safety is established, they went straight to phase-II clinical trials. It appears that they are either ramping up recruitment of patients, or ramping up PR of the trial. Recently there have been very similar "public interest / human face" type newspaper articles on this trial, links below:

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This blog discusses cures and preventatives for type-1 diabetes that are either in human trials or just about to start. Treatments for diabetes are not generally discussed here, unless they can turn into a cure or a preventative. My definition of a cure is this:1. Blood sugar control without testing and with doctor's visits 4 times a year, or less. Any cure must result in an average lifespan close to normal.2. Does not require a lifetime of immunsuppressive drugs, so it is not trading one treatment for another. (but a couple of operations, or a short course of drugs is OK)Obviously, this is my personal definition of a cure; yours may differ.Because a cure for type-1 diabetes is likely to involve a combination of several different drugs or treatments, I try to follow research into anything which may be an important part of the cure.

My Non-Conflict of Interest Statement

I don't work for a company involved in medical research; I never have.

I don't get paid in any way by any company doing medical research; I never have. And that includes free samples, free travel, or free anything.

None of the hours that I have put into my blog, or the posts that I make to any web site, has ever been paid for, nor have I gotten anything free. (Except for some very nice and heart felt thank-you emails, and those are worth more than money.)

My daughter has type-1 diabetes and participates in clinical trials. I sometimes report on trials that she participates in.